[Federal Register Volume 89, Number 76 (Thursday, April 18, 2024)]
[Notices]
[Pages 27767-27769]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-08293]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2023-N-4804]


Agency Information Collection Activities; Submission for Office 
of Management and Budget Review; Comment Request; Expedited Programs 
for Serious Conditions--Drugs and Biologics

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) is announcing that a 
proposed collection of information has been submitted to the Office of 
Management and Budget (OMB) for review and clearance under the 
Paperwork Reduction Act of 1995.

DATES: Submit written comments (including recommendations) on the 
collection of information by May 20, 2024.

ADDRESSES: To ensure that comments on the information collection are 
received, OMB recommends that written comments be submitted to https://www.reginfo.gov/public/do/PRAMain. Find this particular information 
collection by selecting ``Currently under Review--Open for Public 
Comments'' or by using the search function. The OMB control number for 
this information collection is 0910-0765. Also include the FDA docket 
number found in brackets in the heading of this document.

FOR FURTHER INFORMATION CONTACT: Amber Sanford, Office of Operations, 
Food and Drug Administration, Three White Flint North, 10A-12M, 11601 
Landsdown St., North Bethesda, MD 20852, 301-796-8867, 
[email protected].

SUPPLEMENTARY INFORMATION: In compliance with 44 U.S.C. 3507, FDA has 
submitted the following proposed collection of information to OMB for 
review and clearance.

Expedited Programs for Serious Conditions--Drugs and Biologics

OMB Control Number 0910-0765--Extension

    This information collection supports regulations governing FDA 
expedited programs for serious conditions. These provisions are set 
forth in 21 CFR part 312, subpart E and are intended to speed the 
availability of new therapies to patients with serious conditions, 
especially when there are no satisfactory

[[Page 27768]]

alternative therapies, while preserving appropriate standards for 
safety and effectiveness. The regulations call for earlier attention to 
drugs that have promise in treating such conditions, including early 
consultation with FDA for sponsors of such products. Respondents to the 
information collection are sponsors of drug or biologic product 
applications submitted to FDA.
    To assist respondents with the information collection, we developed 
Agency guidance entitled ``Guidance for Industry Expedited Programs for 
Serious Conditions--Drugs and Biologics'' (May 2014). The guidance is a 
resource for information on FDA's policies and procedures related to 
the following expedited programs for serious conditions: (1) fast track 
designation; (2) breakthrough therapy designation; (3) accelerated 
approval; and (4) priority review designation, and describes threshold 
criteria generally applicable to expedited programs, including what is 
meant by serious condition, unmet medical need, and available therapy. 
The guidance addresses the applicability of expedited programs to rare 
diseases, clarification on available therapy, and additional detail on 
possible flexibility in manufacturing and product quality. It also 
clarifies the qualifying criteria for breakthrough therapy designation, 
provides examples of surrogate endpoints and intermediate clinical 
endpoints used to support accelerated approval, and priority review.
    In addition, we developed Agency guidance entitled ``Expedited 
Programs for Regenerative Medicine Therapies for Serious Conditions,'' 
(February 2019) describing the criteria for participation in the 
Regenerative Medicine Advanced Therapy (RMAT) program. The RMAT 
expedited program was approved as part of the 21st Century CURES Act, 
signed December 13, 2016. An RMAT product is intended to treat, modify, 
reverse, or cure serious or life-threatening diseases or conditions, 
and preliminary clinical evidence indicate that the drug has the 
potential to address unmet medical needs for such diseases or 
conditions. This is a Center Biologics Evaluation and Research (CBER) 
program and is included as an expedited program available for serious 
conditions.
    For a sponsor or applicant who seeks fast track, priority, 
breakthrough, RMAT or accelerated approval designation review, approval 
is required to submit a request showing that the drug product: (1) is 
intended for a serious or life-threatening condition and (2) has the 
potential to address an unmet medical need, demonstrate substantial 
improvement over available therapy, or fill an unmet need to be 
approved based on a surrogate endpoint. We expect that most information 
to support a designation request will have been gathered under existing 
requirements for preparing an investigational new drug (IND), new drug 
application (NDA), or biologics license application (BLA). If such 
information has already been submitted to us, the information may be 
summarized in the designation request. A designation request should 
include, where applicable, additional information not specified 
elsewhere by statute or regulation. For example, additional information 
may be needed to show that a product has the potential to address an 
unmet medical need where an approved therapy exists for the serious or 
life-threatening condition to be treated. Such information may include 
clinical data, published reports, summaries of data and reports, and a 
list of references. The amount of information and discussion in a 
designation request should be sufficient to permit a reviewer to assess 
whether the criteria for fast track, priority, breakthrough, RMAT or 
accelerated approval designation have been met.
    After we make an expedited programs designation, a sponsor or 
applicant may submit a premeeting package that may include additional 
information supporting a request to participate in certain expedited 
programs. The premeeting package serves as background information for 
the meeting and should support the intended objectives of the meeting. 
As with the request for expedited programs designation, we expect that 
most sponsors or applicants will have gathered such information to meet 
existing requirements for preparing an IND, NDA, or BLA. These may 
include descriptions of clinical safety and efficacy trials not 
conducted under an IND (e.g., foreign studies) and information to 
support a request for accelerated approval. If such information has 
already been submitted to us, the information may be summarized in the 
premeeting package.
    The guidance documents are available on our website at www.fda.gov/regulatory-information/search-fda-guidance-documents and were issued 
consistent with our good guidance practice regulations in 21 CFR 
10.115, which provide for public comment at any time.
    In the Federal Register of January 9, 2024 (89 FR 1101), FDA 
published a 60-day notice requesting public comment on the proposed 
collection of information. Although one comment was received, it was 
not responsive to the four collection of information topics solicited.
    FDA estimates the burden of this collection of information as 
follows:

                                 Table 1--Estimated Annual Reporting Burden \1\
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                                                         Number of
              Activity                   Number of     responses per   Total annual   Average burden     Total
                                        respondents     respondent       responses     per response      hours
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CDER:
    Priority Review Designation                   81            1.53             124              30       3,720
     Requests (Expedited Programs
     for Serious Conditions Guidance
     (EPSC) Section VIII)...........
    Breakthrough Therapy Designation              71            1.08              77              70       5,390
     Requests (EPSC Section VI).....
    Fast Track Designation Requests              235            1.18             277              60      16,620
     (EPSC Section V)...............
    Accelerated Approval Designation              26            1.27              33             100       3,300
     (EPSC Section VII).............
    Premeeting Packages (21 CFR                  163            1.01             165             100      16,500
     312.82)........................
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        CDER Subtotal...............  ..............  ..............             676  ..............      45,530
CBER:
    Priority Review Designation                    8               1               8              30         240
     Request (EPSC Section VIII)....
    Breakthrough Therapy Designation              15             1.1              17              70       1,190
     Request (EPSC Section VI)......
    Fast Track Designation Requests               64             1.2              77              60       4,620
     (EPSC Section VII).............
    RMAT Designation Requests                     33             1.1              36              60       2,160
     (Regenerative Medicine
     Therapies for Serious
     Conditions Guidance (RMAT)
     Section III)...................
    Premeeting Packages (RMAT                    146             1.9             277             100      27,700
     Section V).....................
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        CBER Subtotal...............  ..............  ..............             415  ..............      35,910
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[[Page 27769]]

 
            Total...................  ..............  ..............           1,091  ..............      81,440
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.

    Based on FY 2022 receipts, we estimate that for Center for Drug 
Evaluation and Research (CDER) products, 81 respondents will submit 124 
requests for priority review designation annually, and we assume 30 
hours are needed to prepare such a request. We estimate 71 respondents 
will submit 77 requests for breakthrough designation annually, and we 
assume 70 hours are needed to prepare such a request. We estimate that 
235 respondents will submit 277 requests for fast-track designation 
requests annually, and we assume 60 hours are required to prepare such 
a request. We estimate 26 respondents will submit 33 accelerated 
approval designation requests annually and we assume 100 hours are 
required to prepare such a request. Finally, CDER received 165 pre-
meeting package submissions from 163 respondents. We assume 100 hours 
are needed to prepare a pre-meeting package.
    Similarly, also based on FY 2022 receipts, we estimate that for 
CBER products, 8 applicants will submit 8 requests for priority review 
designation annually, and we assume 30 hours are required to prepare 
such a request. We estimate 15 respondents will submit 17 requests for 
breakthrough designation annually, and we assume 70 hours are needed to 
prepare such a request. We estimate that 64 respondents will submit 78 
requests for fast-track designation annually, and we assume 60 hours is 
required to prepare such a request. We also estimate 33 respondents 
will submit 35 requests for RMAT designation annually and assume that 
60 hours are needed to prepare each RMAT designation request. Finally, 
CBER received 283 pre-meeting package submissions from 146 respondents. 
We assume 100 hours are needed to prepare a pre-meeting package.
    Based on a review of the information collection since our last 
request for OMB approval, we have increased our burden estimate by 143 
responses and 10,350 hours to reflect actual submissions we have 
received. We attribute these changes to increased interest in the 
expedited programs, new expedited programs, and an increase in the 
number of submissions we received over the last few years.

    Dated: April 15, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-08293 Filed 4-17-24; 8:45 am]
BILLING CODE 4164-01-P